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Victory for Rare Disease Patients: North Carolina Expands the Right to Try

July 9, 2024

In a life-changing victory for patients with rare and ultra-rare diseases, North Carolina Governor Roy Cooper yesterday signed the Goldwater Institute’s Right to Try Individualized Treatments Act. The innovative, nonpartisan reform offers hope to those who need it most, allowing suffering patients to work directly with their physician to safely seek the most groundbreaking of investigational personalized care options.

Championed by Representative Allen Chesser and Senator Benton Sawrey and unanimously approved by both houses of the North Carolina General Assembly, House Bill 98 ensures that patients suffering from severely debilitating or life-threatening illnesses can participate in the latest and most promising paradigm shift in medicine—individualized treatments that are tailor-made for each patient based on their unique genetic information.

For too long, the Food and Drug Administration (FDA) has sidelined the needs of rare disease patients by blocking or delaying access to the most innovative personalized treatments. Indeed, 95% of rare diseases have no FDA-approved treatment.

The federal government’s decades-old clinical trial evaluation system is designed for treatments meant for large patient populations—not an individual patient. As a result, individualized treatments are still subject to the same “one-size-fits-all” clinical trial process as drugs intended for thousands upon thousands of patients, which delays or denies access to potentially lifesaving treatments for millions of rare disease patients.

Consider the Riley family of Arizona, whose story inspired Goldwater’s Right to Try for Individualized Treatments reform. After baby Keira Riley was diagnosed with a rare and fatal genetic brain disease, her family found out about a specialized type of gene therapy could help save her life. But since it wasn’t available in the U.S. due to FDA restrictions, the Rileys had to uproot their family, leave their lives and support network behind in Arizona, and move to Italy to obtain the treatment. Countless Americans across the country have faced similar heart-wrenching decisions and undertaken drastic measures to ensure their loved ones receive treatment.

“Individualized treatments are being pioneered all over the world. Far too often, U.S. patients are forced to travel overseas at their own expense for potentially lifesaving treatments due to archaic federal regulations, or else they succumb to their cruel rare disease,” Goldwater Institute Senior Fellow Naomi Lopez said. “With House Bill 98, the North Carolina General Assembly made the Tar Heel State a leader in ensuring that the right treatment gets to the right patient at the right time. North Carolinians now have the option of seeking the most innovative and promising treatments in Raleigh rather than Rome.”

The legislation builds on North Carolina’s legacy of biotechnology innovation in the Research Triangle and represents the next step in the nationwide nonpartisan movement to enact this landmark Right to Try expansion. A longtime hub for advancements in biotechnology and healthcare, North Carolina is no stranger to protecting a patient’s right to save their own life. In 2015, North Carolina passed the Goldwater Institute’s original Right to Try Act, sponsored by Rep. Hugh Blackwell, to give terminally ill patients access to potentially lifesaving treatments that have completed a Phase One clinical trial.

North Carolina joins a growing list of states that have expanded the Right to Try to include individualized treatments. In 2024 alone, Louisiana, Maryland and Mississippi passed similar laws, joining Arizona and Nevada, who enacted this crucial reform in 2022 and 2023, respectively.

The federal government’s inaction and indifference means that far too many rare disease patients across the country are being denied access to individualized treatments that offer a glimmer of hope. Their suffering is an ongoing reminder that every state across our nation must follow in North Carolina’s footsteps by passing the Right to Try for Individualized Treatments.

The Goldwater Institute thanks Rep. Chesser, Sen. Sawrey, and the many advocates who voiced support for this landmark legislation. The Institute would also like to thank Rep. Blackwell, who spearheaded the original Right to Try Act in North Carolina back in 2015, as well as Governor Roy Cooper and the entire General Assembly for unanimously protecting the right to save one’s own life.

Brian Norman is the Director of State Affairs at the Goldwater Institute, where he assists in the development and implementation of Goldwater’s national legislative affairs strategy.

 

 

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